Newsletter Xagena

New data from the core and extension phases of the DRIVE PK phase 2 study of Mitapivat ( AG-348 ) in adults with pyruvate kinase ( PK ) deficiency were published in the New England Journal of Medicine ...

The New England Journal of Medicine ( NEJM ) has published the results from a phase 2 dose-finding and extension study for Vosoritide, an investigational analog of C-type natriuretic peptide ( CNP ), ...

The U.S. Food and Drug Administration ( FDA ) has approved Zolgensma ( Onasemnogene abeparvovec-xioi ), the first gene therapy approved to treat children less than two years of age with spinal muscula ...

Novo Nordisk has announced the submission of a Biologics License Applications ( BLA ) to the US Food and Drug Administration ( FDA ) and a Marketing Authorisation Application ( MAA ) to the European M ...

The results of a pediatric phase 2 clinical trial of Burosumab ( Crysvita ) for the treatment of X-linked hypophosphatemia ( XLH ) in children aged 5 to 12 years were published by the New England Jour ...

Amyotrophic lateral sclerosis ( ALS ) is a rapidly progressing neurodegenerative disease that is characterized by motor neuron loss and that leads to paralysis and death 2-5 years after disease onset. ...

The FDA ( U.S. Food and Drug Administration ) has approved Spinraza ( Nusinersen ), the first drug approved to treat children and adults with spinal muscular atrophy ( SMA ), a rare and often fatal ge ...

Results from a phase 2 clinical trial investigating Palovarotene for the treatment of fibrodysplasia ossificans progressiva ( FOP ) were presented.
FOP is an extremely rare, serious disease in which ...

The positive results of the first pivotal phase 3 study of the investigational medicine Epidiolex ( Cannabidiol or CBD ) for the treatment of Dravet syndrome were announced.
In this study, Epidiolex ...